The impact of amortization of gene therapies funding on the results and conclusions of CEMs and BIMs

ABSTRACTBackground: Gene replacement therapy (GRT) is a treatment method used to combat or prevent various diseases. Its high one-off cost constitutes a major obstacle for successful market access. This paper aims to assess and discuss the applicability of amortization in models, such as cost-effect...

Full description

Saved in:
Bibliographic Details
Main Authors: Hubert Polek, Justyna Janik, Ewelina Paterak, Monique Dabbous, Michał Pochopień, Mondher Toumi
Format: Article
Language:English
Published: MDPI AG 2023-12-01
Series:Journal of Market Access & Health Policy
Subjects:
Online Access:https://www.tandfonline.com/doi/10.1080/20016689.2023.2232648
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1850234398549475328
author Hubert Polek
Justyna Janik
Ewelina Paterak
Monique Dabbous
Michał Pochopień
Mondher Toumi
author_facet Hubert Polek
Justyna Janik
Ewelina Paterak
Monique Dabbous
Michał Pochopień
Mondher Toumi
author_sort Hubert Polek
collection DOAJ
description ABSTRACTBackground: Gene replacement therapy (GRT) is a treatment method used to combat or prevent various diseases. Its high one-off cost constitutes a major obstacle for successful market access. This paper aims to assess and discuss the applicability of amortization in models, such as cost-effectiveness models (CEMs) and budget impact models (BIMs) informing HTA recommendations and reimbursement decisions.Methods and findings: A hypothetical CEA and BIA were considered. The objective was to compare the GRT with and without amortization. A straight-line amortization model was used. The CEM and BIM were considered and assessed based on two set of scenarios: considering different amortization duration or different discounting rate. The impact of amortization against the total cost of gene therapy was assessed for all the scenarios.The cost difference between GRT with and without amortization in relation to its total cost was -$58,855, thus amortization does not have a significant impact on the results and conclusions of the cost-effectiveness analysis. For BIM in the base case, amortization had no impact on the results.Conclusion: Amortization has negligible impact on the results of CEM and total BIM and no impact on the conclusions from the model. One exception is the budget impact in case of an amortization period longer than the time horizon of BIM, where a half of the GRT price is moved beyond the model time horizon. Amortization has a distinguishing effect from an accounting perspective, but it does not have any implication for payers.
format Article
id doaj-art-b0d9fcd2aab04c4a88bf0a59c90de313
institution OA Journals
issn 2001-6689
language English
publishDate 2023-12-01
publisher MDPI AG
record_format Article
series Journal of Market Access & Health Policy
spelling doaj-art-b0d9fcd2aab04c4a88bf0a59c90de3132025-08-20T02:02:39ZengMDPI AGJournal of Market Access & Health Policy2001-66892023-12-0111110.1080/20016689.2023.2232648The impact of amortization of gene therapies funding on the results and conclusions of CEMs and BIMsHubert Polek0Justyna Janik1Ewelina Paterak2Monique Dabbous3Michał Pochopień4Mondher Toumi5Department of Health Economics and Outcomes Research, Assignity, Cracow, PolandDepartment of Health Economics and Outcomes Research, Putnam PHMR, Cracow, PolandDepartment of Health Economics and Outcomes Research, Putnam PHMR, Cracow, PolandDépartement de Santé Publique, Aix-Marseille Université, Marseille, FranceDépartement de Santé Publique, Aix-Marseille Université, Marseille, FranceDépartement de Santé Publique, Aix-Marseille Université, Marseille, FranceABSTRACTBackground: Gene replacement therapy (GRT) is a treatment method used to combat or prevent various diseases. Its high one-off cost constitutes a major obstacle for successful market access. This paper aims to assess and discuss the applicability of amortization in models, such as cost-effectiveness models (CEMs) and budget impact models (BIMs) informing HTA recommendations and reimbursement decisions.Methods and findings: A hypothetical CEA and BIA were considered. The objective was to compare the GRT with and without amortization. A straight-line amortization model was used. The CEM and BIM were considered and assessed based on two set of scenarios: considering different amortization duration or different discounting rate. The impact of amortization against the total cost of gene therapy was assessed for all the scenarios.The cost difference between GRT with and without amortization in relation to its total cost was -$58,855, thus amortization does not have a significant impact on the results and conclusions of the cost-effectiveness analysis. For BIM in the base case, amortization had no impact on the results.Conclusion: Amortization has negligible impact on the results of CEM and total BIM and no impact on the conclusions from the model. One exception is the budget impact in case of an amortization period longer than the time horizon of BIM, where a half of the GRT price is moved beyond the model time horizon. Amortization has a distinguishing effect from an accounting perspective, but it does not have any implication for payers.https://www.tandfonline.com/doi/10.1080/20016689.2023.2232648Gene therapiesamortizationeconomic analysisaffordabilityassessment
spellingShingle Hubert Polek
Justyna Janik
Ewelina Paterak
Monique Dabbous
Michał Pochopień
Mondher Toumi
The impact of amortization of gene therapies funding on the results and conclusions of CEMs and BIMs
Journal of Market Access & Health Policy
Gene therapies
amortization
economic analysis
affordability
assessment
title The impact of amortization of gene therapies funding on the results and conclusions of CEMs and BIMs
title_full The impact of amortization of gene therapies funding on the results and conclusions of CEMs and BIMs
title_fullStr The impact of amortization of gene therapies funding on the results and conclusions of CEMs and BIMs
title_full_unstemmed The impact of amortization of gene therapies funding on the results and conclusions of CEMs and BIMs
title_short The impact of amortization of gene therapies funding on the results and conclusions of CEMs and BIMs
title_sort impact of amortization of gene therapies funding on the results and conclusions of cems and bims
topic Gene therapies
amortization
economic analysis
affordability
assessment
url https://www.tandfonline.com/doi/10.1080/20016689.2023.2232648
work_keys_str_mv AT hubertpolek theimpactofamortizationofgenetherapiesfundingontheresultsandconclusionsofcemsandbims
AT justynajanik theimpactofamortizationofgenetherapiesfundingontheresultsandconclusionsofcemsandbims
AT ewelinapaterak theimpactofamortizationofgenetherapiesfundingontheresultsandconclusionsofcemsandbims
AT moniquedabbous theimpactofamortizationofgenetherapiesfundingontheresultsandconclusionsofcemsandbims
AT michałpochopien theimpactofamortizationofgenetherapiesfundingontheresultsandconclusionsofcemsandbims
AT mondhertoumi theimpactofamortizationofgenetherapiesfundingontheresultsandconclusionsofcemsandbims
AT hubertpolek impactofamortizationofgenetherapiesfundingontheresultsandconclusionsofcemsandbims
AT justynajanik impactofamortizationofgenetherapiesfundingontheresultsandconclusionsofcemsandbims
AT ewelinapaterak impactofamortizationofgenetherapiesfundingontheresultsandconclusionsofcemsandbims
AT moniquedabbous impactofamortizationofgenetherapiesfundingontheresultsandconclusionsofcemsandbims
AT michałpochopien impactofamortizationofgenetherapiesfundingontheresultsandconclusionsofcemsandbims
AT mondhertoumi impactofamortizationofgenetherapiesfundingontheresultsandconclusionsofcemsandbims